Sanofi’s Pompe disease enzyme therapy clears Phase 3 in infants, opening US label expansion

Sanofi announced that its next‑generation enzyme replacement therapy for Pompe disease met its primary endpoints in a registrational Phase 3 trial involving infants with the severe infantile‑onset form of the disorder.

The study demonstrated that the therapy improved key clinical measures compared with standard of care, supporting its potential to become a new treatment option for this high‑need patient population.

Successful results pave the way for Sanofi to seek an expanded label in the United States, which could broaden access to the drug for affected infants and represent a significant commercial opportunity for the company.